Denali Therapeutics Announces Continued Progress in DNL310 (ETV:IDS) Program for MPS II (Hunter Syndrome) Supporting Planned Initiation of Phase 2/3 Clinical Trial

• Longer-term data in 20 patients show sustained normalization to healthy levels of CSF heparan sulfate and improvements in markers of lysosomal function consistent with durable CNS activity, now with up to one year of intravenous dosing with DNL310
• Safety profile with up to 56 weeks of dosing remains consistent with standard-of-care enzyme replacement therapy
• Data continue to support initiation of dosing with DNL310 in a potentially registrational Phase 2/3 clinical trial expected to begin in 1H 2022
• These longer-term data with DNL310 continue to provide biomarker proof of concept for Denali’s Transport Vehicle (TV) platform to deliver biotherapeutics across the blood-brain barrier

Excerpt from the Press Release:

SOUTH SAN FRANCISCO, Calif., Feb. 10, 2022 (GLOBE NEWSWIRE) — Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases, today announced that new longer-term data from an ongoing Phase 1/2 clinical trial of DNL310 (ETV:IDS) are being presented today at WORLDSymposium™. DNL310 is an investigational brain-penetrant enzyme replacement therapy intended to treat both central nervous system (CNS) and peripheral manifestations of MPS II (Hunter syndrome). 

“The longer-term Phase 1/2 data, now up to approximately one year for Cohort A and six months for Cohort B, support DNL310 as a differentiated enzyme replacement therapy that rapidly normalizes CSF heparan sulfate and maintains improvement in CSF biomarkers of lysosomal function with weekly intravenous dosing,” said Carole Ho, M.D., Denali’s Chief Medical Officer. “With additional patient data now available, we are also encouraged that we continue to see improvements in exploratory clinical outcomes in the majority of individuals as assessed after six months of open label DNL310 treatment. These data continue to support initiation of a potentially registrational Phase 2/3 study of DNL310 in the first half of 2022. We look forward to continued collaboration with the MPS II community to advance research and DNL310 as a potential treatment for affected individuals and their families.”

Phase 1/2 study data being presented at WORLDSymposium™ today at 8 a.m. Pacific Time

The Phase 1/2 study data being presented at WORLDSymposium™ today include longer-term biomarker data up to Weeks 49 and 24 from Cohort A and B, respectively; clinical outcomes data across Cohorts A and B at Week 24; and safety data up to Weeks 56 and 39 from Cohorts A and B, respectively. All patients have neuronopathic MPS II disease except for one patient with non-neuronopathic MPS II disease in Cohort B. The median age of patients is 6 years in both cohorts, with the youngest patients aged 5 and 2 in Cohorts A and B, respectively.

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